Knockout of the CEP290 gene in human induced pluripotent stem cells

CEP290 is a principal component of the primary cilium and is important for the proper function of ciliated cells. CEP290 mutations have been linked to numerous ciliopathies, with a wide range of phenotypic severities, but with poor genotype:phenotype correlation. Here we have used CRISPR/Cas9 techno...

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Detalles Bibliográficos
Autores principales: Fogerty, Joseph, Perkins, Brian D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8127132/
https://www.ncbi.nlm.nih.gov/pubmed/33626495
http://dx.doi.org/10.1016/j.scr.2021.102243
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author Fogerty, Joseph
Perkins, Brian D.
author_facet Fogerty, Joseph
Perkins, Brian D.
author_sort Fogerty, Joseph
collection PubMed
description CEP290 is a principal component of the primary cilium and is important for the proper function of ciliated cells. CEP290 mutations have been linked to numerous ciliopathies, with a wide range of phenotypic severities, but with poor genotype:phenotype correlation. Here we have used CRISPR/Cas9 technology to target the CEP290 gene and generate a line of induced pluripotent stem cells that lack detectable CEP290 expression, but retain a normal karyotype and differentiation potential. This line of cells will be useful for the study of disorders resulting from CEP290 mutations.
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spelling pubmed-81271322021-05-17 Knockout of the CEP290 gene in human induced pluripotent stem cells Fogerty, Joseph Perkins, Brian D. Stem Cell Res Article CEP290 is a principal component of the primary cilium and is important for the proper function of ciliated cells. CEP290 mutations have been linked to numerous ciliopathies, with a wide range of phenotypic severities, but with poor genotype:phenotype correlation. Here we have used CRISPR/Cas9 technology to target the CEP290 gene and generate a line of induced pluripotent stem cells that lack detectable CEP290 expression, but retain a normal karyotype and differentiation potential. This line of cells will be useful for the study of disorders resulting from CEP290 mutations. 2021-02-11 2021-04 /pmc/articles/PMC8127132/ /pubmed/33626495 http://dx.doi.org/10.1016/j.scr.2021.102243 Text en https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) ).
spellingShingle Article
Fogerty, Joseph
Perkins, Brian D.
Knockout of the CEP290 gene in human induced pluripotent stem cells
title Knockout of the CEP290 gene in human induced pluripotent stem cells
title_full Knockout of the CEP290 gene in human induced pluripotent stem cells
title_fullStr Knockout of the CEP290 gene in human induced pluripotent stem cells
title_full_unstemmed Knockout of the CEP290 gene in human induced pluripotent stem cells
title_short Knockout of the CEP290 gene in human induced pluripotent stem cells
title_sort knockout of the cep290 gene in human induced pluripotent stem cells
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8127132/
https://www.ncbi.nlm.nih.gov/pubmed/33626495
http://dx.doi.org/10.1016/j.scr.2021.102243
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